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Mucopolysaccharidosis I under enzyme replacement therapy with laronidase—A mortality case with autopsy report
Author(s) -
Lin H.Y.,
Lin S.P.,
Chuang C.K.,
Chen M.R.,
Chen B.F.,
Wraith J. E.
Publication year - 2005
Publication title -
journal of inherited metabolic disease
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.462
H-Index - 102
eISSN - 1573-2665
pISSN - 0141-8955
DOI - 10.1007/s10545-005-0211-x
Subject(s) - enzyme replacement therapy , autopsy , medicine , mucopolysaccharidosis , medical therapy , human genetics , pediatrics , cause of death , surgery , disease , biology , biochemistry , gene
There is little information about MPS I‐related complications during laronidase therapy. We describe the first autopsy report of a young male MPS I patient who died of infection‐induced cardiopulmonary failure following 2 years of weekly treatment with laronidase.